Gene therapy being developed as a one-time treatment to address the underlying cause of the disease by inserting a functional copy of the human IDUA gene into a patient’s own hematopoietic stem cells HURCULES study to enroll 40 patients at clinical sites across the U.S. and Europe TOKYO, LONDON and BOSTON, Feb. 05, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today an

Three oral presentations and seven posters highlight transformative potential of HSC gene therapy and newborn screening to address severe neurometabolic diseasesBOSTON and LONDON, Feb. 02, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced 10 presentations from across its neurometabolic portfolio will be featured at the 20th Annu

BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD). The agreement covers several member countries, including Belgium, The Netherlands, and

Acquisition enriches Kyowa Kirin’s portfolio, enables the development of numerous promising candidates with a clinically differentiated platform, and helps to resource ongoing and future launches of Libmeldy® (atidarsagene autotemcel) in early-onset MLDTOKYO and LONDON and BOSTON, Jan. 24, 2024 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnolo

BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD). MLD is a rare, fatal genetic disorder caused by a mutation in the gene responsible for encoding the enzyme arylsulfatase A (ARSA) leading to neu

Previously reported results from ongoing proof-of-concept study showed extensive metabolic correction, continued cognitive, motor, and physical development, as well as early improvements in skeletal health Additionally, study investigators presented favorable outcomes for other disease manifestations not effectively addressed by the current standard of care at ESGCT 2023 Global registrational trial expected to commence by year-end BOSTON and LONDON, Nov. 30, 2023 (GLOBE NEWSWIRE) -- Orchard Ther

ORTX Sees Libmeldy Revenue Growth and Prepares for Potential U.S. Launch in 2024

Reported $5.6 million in quarterly Libmeldy net revenue and $12.7 million year-to-date Planned acquisition by Kyowa Kirin valued at up to $477.6 million with an anticipated closing in the first quarter of 2024 Biologics License Application for OTL-200 in MLD accepted by U.S. FDA under Priority Review; PDUFA date set for March 18, 2024 Ten prospective newborn screening studies are active globally with approximately 200,000 babies screened Eight presentations at ESGCT highlighted the differentiate

Arvinas, Inc. (ARVN) was a big mover last session on higher-than-average trading volume. The latest trend in earnings estimate revisions might not help the stock continue moving higher in the near term.

New clinical data beyond previously reported neurological and skeletal results demonstrate favorable outcomes for multiple disease manifestations not effectively addressed by the current standard of careBOSTON and LONDON, Oct. 26, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced a range of interim clinical outcomes, in addition to the previously reported neurological and skeletal results, from the company’s ongoing proof-of-concept (PoC)